Strong Community Support Essential to Passage of Critical Muscular Dystrophy Legislation

Centers of research excellence. Clinical trials. New insights into disease causation and progression. Optimal patient care guidelines. A robust pipeline of promising research.

These are the tangible results of the MD CARE Act (H.R. 594/S. 315) — and these are what advocates are seeking to preserve and expand in MDA's all-out push that began Feb. 8, 2013, to convince Congress to reauthorize this critical legislation for muscular dystrophy.

(To read the Senate and House bill text, visit thomas.loc.gov and type in the bill number in the search box.)

Since the original passage of the MD CARE Act in 2001, “there have been 67 clinical trials of drugs or therapies for the muscular dystrophies, with 37 clinical trials currently under way,” says Annie Kennedy, MDA senior vice president for advocacy.

“We’ve ‘moved the needle’ in muscular dystrophy care and research. We must keep the momentum going strong.”

Although a number of congressional champions strongly support reauthorization of the Act, passage is far from guaranteed in the current political climate. Nonetheless, MDA — in concert with Parent Project Muscular Dystrophy and the Foundation to Eradicate Duchenne — has helped to shape the 2013 reauthorization bill so that it expands on a decade of progress and continues the push toward the ultimate goal: improved health outcomes and effective, available treatments for people with muscular dystrophy.

MD CARE: A brief history

At the turn of the new millennium, total governmental funding for muscular dystrophy research was $17 million, there were no new drugs in development, there were no care guidelines and no patient registries, and the average life span for someone with Duchenne muscular dystrophy (DMD) was the late teens.

In 2001, after extensive lobbying by the muscular dystrophy community (including congressional testimony by former MDA National Chairman Jerry Lewis and former National Goodwill Ambassador Ben Cumbo), Congress passed the Muscular Dystrophy Community Assistance, Research and Education (MD CARE) Act.

The Act provided essential government funding for the infrastructure needed to accelerate research and bring drugs to market for all nine forms of muscular dystrophy. 

The ensuing decade saw an unprecedented national focus on coordinating and intensifying MD research:

• The Centers for Disease Control and Prevention (CDC) launched MD STARnet (Muscular Dystrophy Surveillance, Tracking and Research Network), a data collection and surveillance project.  
• The National Institutes of Health (NIH) established nine specialized muscular dystrophy research centers around the U.S., called Paul D. Wellstone Muscular Dystrophy Cooperative Research Centers, or “Centers of Excellence.” (The late Minnesota Senator Paul Wellstone, a founding champion of the MD CARE Act, died in a plane crash in 2002.)
• A public-private committee, the Muscular Dystrophy Coordinating Committee (MDCC), was created to expand, strengthen and coordinate muscular dystrophy research and care efforts. Under the direction of the U.S. Secretary of Health and Human Services, representatives from a number of patient advocacy organizations (including MDA) and from various federal agencies identified gaps in existing research and care programs, and looked for opportunities for collaboration among the committee members.

Today, total governmental funding for muscular dystrophy research has swelled to $75 million annually.  Nine new drugs are in trial for DMD and almost a dozen others in preclinical development. Care guidelines for DMD have been disseminated worldwide, and guidelines are in development for facioscapulohumeral muscular dystrophy (FSHD), myotonic muscular dystrophy (MMD), limb-girdle muscular dystrophy (LGMD) and congenital muscular dystrophy (CMD).

And in just over a decade, life span in DMD had increased by 10 years.

MD CARE reauthorization bill expands on progress

The 2013 reauthorization bill was introduced in the U.S. Senate (S. 315) on Feb. 13, 2013 by Senators Amy Klobuchar (D-Minn.), Roger Wicker (R-Miss.), Bernie Sanders (I-Vt.), Susan Collins (R-Maine), Robert Menendez (D-N.J.), Johnny Isakson (R-Ga.), Barbara Mikulski (D-Md.), Patrick Leahy (D-Vt.), Frank Lautenberg (D-N.J.) and Bill Nelson (D-Fla.).

The bill was introduced in the U.S. House of Representatives (H.R. 594) on Feb. 8, 2013, by co-sponsors Eliot Engel (D-N.Y.) and Michael Burgess (R-Texas), who also spearheaded the the reauthorization in 2008.

It seeks to expand the scope of the bill in several ways, including:

• expand the eligible fields of research at the Wellstone Centers to include cardiac and pulmonary function;
• require that the NIH support no fewer than eight Wellstone Centers at any given time;
• direct the CDC to update — and widely share with health care professionals and the general public — existing care considerations for DMD/BMD (also known as DBMD);
• direct the CDC to develop care considerations for adults with DMD/BMD; and
• develop/distribute acute care considerations for all forms of muscular dystrophy.

If passed, the bill also would expand the mission of the MDCC (the public-private coordinating committee) to include:

• developing a plan for conducting and supporting muscular dystrophy research and education, such as developing optimal clinical care interventions for young adults with DMD/BMD, and demonstrating the cost-effectiveness of providing independent living resources and support services for young adults with all forms of muscular dystrophy;
• developing a plan for speeding government approval of emerging therapies and personalized medicines for people with muscular dystrophy; and
• evaluating ways to enhance the current clinical research infrastructure that is required to test emerging therapies, including expanding the use of regulatory science and facilitating cooperation and communication across federal agencies.

Strong community support essential to passage

MDA is at the forefront of the effort to convince Congress to reauthorize and allocate funding for the MD CARE Act. Advocacy by the muscular dystrophy community is key to this effort.

“The MD CARE Act has been a ‘game changer’ for our MD community,” Kennedy says. “We must ensure that our federal research funding remains robust, that we optimize our clinical trial infrastructure, and that we are able to deliver effective therapies to our loved ones who await them. We need the active outreach of every member of the MDA community — every clinician, researcher, family, sponsor, volunteer, advocate — to ensure legislative success.

“Our community has overcome amazing challenges before, and we will do it again in 2013. Please ‘Take 5’ and contact your elected officials to urge their support of the MD CARE Act of 2013 and keep our lifesaving research and care momentum strong.”

For more information

To learn more about MDA's efforts to reauthorize the MD CARE Act of 2013, see:

• MDA and the MD CARE Act;
• MD-CARE Passes (information about the first reauthorization of the bill, Quest, November 2008); and
• Mr. Lewis Goes to Washington (MDA testimony to Congress about the original MD CARE Act, Quest, April 2001).

And be sure to follow MDA on Facebook and Twitter to stay in touch with the latest news on the 2013 reauthorization legislation.